TWI823830B - 用於ａ型血友病基因治療之編碼表現增加之重組ｆｖｉｉｉ變異體之病毒載體 - Google Patents

用於ａ型血友病基因治療之編碼表現增加之重組ｆｖｉｉｉ變異體之病毒載體 Download PDF

Info

Publication number
TWI823830B
TWI823830B TW105136967A TW105136967A TWI823830B TW I823830 B TWI823830 B TW I823830B TW 105136967 A TW105136967 A TW 105136967A TW 105136967 A TW105136967 A TW 105136967A TW I823830 B TWI823830 B TW I823830B
Authority
TW
Taiwan
Prior art keywords
seq
factor viii
sequence
polynucleotide
nucleotide sequence
Prior art date
Application number
TW105136967A
Other languages
English (en)
Chinese (zh)
Other versions
TW201723180A (zh
Inventor
法科－甘特 福克諾
法蘭西斯卡 霍林
喬安斯 藍格勒
漢斯彼得 羅騰史戴納
弗利茲 許夫利格
Original Assignee
日商武田藥品工業股份有限公司
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by 日商武田藥品工業股份有限公司 filed Critical 日商武田藥品工業股份有限公司
Publication of TW201723180A publication Critical patent/TW201723180A/zh
Application granted granted Critical
Publication of TWI823830B publication Critical patent/TWI823830B/zh

Links

Images

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/745—Blood coagulation or fibrinolysis factors
    • C07K14/755—Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14142—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/007—Vectors comprising a special translation-regulating system cell or tissue specific

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Hematology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Immunology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Diabetes (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
TW105136967A 2015-11-13 2016-11-11 用於ａ型血友病基因治療之編碼表現增加之重組ｆｖｉｉｉ變異體之病毒載體 TWI823830B (zh)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562255323P 2015-11-13 2015-11-13
US62/255,323 2015-11-13

Publications (2)

Publication Number Publication Date
TW201723180A TW201723180A (zh) 2017-07-01
TWI823830B true TWI823830B (zh) 2023-12-01

Family

ID=57539606

Family Applications (1)

Application Number Title Priority Date Filing Date
TW105136967A TWI823830B (zh) 2015-11-13 2016-11-11 用於ａ型血友病基因治療之編碼表現增加之重組ｆｖｉｉｉ變異體之病毒載體

Country Status (17)

Country Link
US (1) US10189889B2 (fr)
EP (1) EP3374388A1 (fr)
JP (1) JP6695426B2 (fr)
KR (1) KR102404550B1 (fr)
CN (1) CN108602876B (fr)
AU (1) AU2016354550B2 (fr)
BR (1) BR112018009732A8 (fr)
CA (1) CA3005565A1 (fr)
CL (2) CL2018001301A1 (fr)
CO (1) CO2018005380A2 (fr)
EA (2) EA036944B1 (fr)
IL (1) IL259295B2 (fr)
MX (1) MX2018005969A (fr)
SG (1) SG11201804064WA (fr)
TW (1) TWI823830B (fr)
WO (1) WO2017083764A1 (fr)
ZA (1) ZA201803442B (fr)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
WO2020150375A1 (fr) * 2019-01-16 2020-07-23 Baxalta Incorporated Vecteurs viraux codant pour des variants fviii de recombinaison avec une expression accrue pour la thérapie génique de l'hémophilie a
SG11202108357PA (en) 2019-02-15 2021-08-30 Crispr Therapeutics Ag Gene editing for hemophilia a with improved factor viii expression
CN109735547B (zh) * 2019-03-19 2022-05-31 青岛蔚蓝生物集团有限公司 一种提高毕赤酵母外源蛋白表达量的启动子及其应用
JP2022537555A (ja) * 2019-06-20 2022-08-26 武田薬品工業株式会社 ウイルスベースの遺伝子療法による治療方法
CN112575034B (zh) * 2019-09-29 2023-04-25 济南赛尔生物科技股份有限公司 一种治疗a型血友病的产品及应用

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20150071883A1 (en) * 2013-09-12 2015-03-12 Biomarin Pharmaceutical Inc. Adeno-Associated Virus Factor VIII Vectors

Family Cites Families (39)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4797368A (en) 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
EP0218712B1 (fr) 1985-04-12 1992-02-26 Genetics Institute, Inc. Nouvelles proteines procoagulantes
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US5595886A (en) 1986-01-27 1997-01-21 Chiron Corporation Protein complexes having Factor VIII:C activity and production thereof
US5543502A (en) 1986-06-24 1996-08-06 Novo Nordisk A/S Process for producing a coagulation active complex of factor VIII fragments
US6060447A (en) 1987-05-19 2000-05-09 Chiron Corporation Protein complexes having Factor VIII:C activity and production thereof
IL86693A (en) 1987-06-12 1994-06-24 Stichting Centraal Lab Proteins that have the activity IIIV of the blood, a process for their preparation that uses cells are produced through genetic engineering and pharmaceutical preparations that contain them
US6346513B1 (en) 1987-06-12 2002-02-12 Baxter Trading Gmbh Proteins with factor VIII activity: process for their preparation using genetically-engineered cells and pharmaceutical compositions containing them
FR2619314B1 (fr) 1987-08-11 1990-06-15 Transgene Sa Analogue du facteur viii, procede de preparation et composition pharmaceutique le contenant
ATE322547T1 (de) 1993-06-10 2006-04-15 Genetic Therapy Inc Adenovirale vektoren für die behandlung der hämophilie
SE504074C2 (sv) 1993-07-05 1996-11-04 Pharmacia Ab Proteinberedning för subkutan, intramuskulär eller intradermal administrering
SE9503380D0 (sv) 1995-09-29 1995-09-29 Pharmacia Ab Protein derivatives
US6013516A (en) 1995-10-06 2000-01-11 The Salk Institute For Biological Studies Vector and method of use for nucleic acid delivery to non-dividing cells
US6458563B1 (en) 1996-06-26 2002-10-01 Emory University Modified factor VIII
US6114148C1 (en) 1996-09-20 2012-05-01 Gen Hospital Corp High level expression of proteins
US5994136A (en) 1997-12-12 1999-11-30 Cell Genesys, Inc. Method and means for producing high titer, safe, recombinant lentivirus vectors
US6924365B1 (en) 1998-09-29 2005-08-02 Transkaryotic Therapies, Inc. Optimized messenger RNA
US6200560B1 (en) 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
US6221349B1 (en) * 1998-10-20 2001-04-24 Avigen, Inc. Adeno-associated vectors for expression of factor VIII by target cells
CN1179976C (zh) * 2000-12-29 2004-12-15 中国科学院上海生物化学研究所 产生凝血因子ⅷ的生产方法和宿主细胞
CA2461443C (fr) 2001-10-05 2011-07-12 Emory University Sequences d'acides nucleiques et d'acides amines codant des polypeptides de facteur viii a expresseur a haut rendement et leurs procedes d'utilisation
ES2975413T3 (es) 2001-12-17 2024-07-05 Univ Pennsylvania Secuencias de serotipo 8 de virus adenoasociado (AAV), vectores que las contienen y usos de las mismas
US7041635B2 (en) 2003-01-28 2006-05-09 In2Gen Co., Ltd. Factor VIII polypeptide
US7943374B2 (en) 2005-08-21 2011-05-17 Markus Hildinger Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb
KR100959454B1 (ko) 2007-12-10 2010-05-25 주식회사 동부하이텍 반도체 소자 및 그 제조 방법
GB0911870D0 (en) 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
US20130017997A1 (en) * 2010-08-19 2013-01-17 Amunix Operating Inc. Factor VIII Compositions and Methods of Making and Using Same
LT2802668T (lt) * 2012-01-12 2018-11-26 Bioverativ Therapeutics Inc. Imunogeninio atsako prieš faktorių viii individuose, kuriems skiriama faktoriaus viii terapija, sumažinimas
RS63870B1 (sr) * 2012-02-15 2023-01-31 Bioverativ Therapeutics Inc Sastavi faktora viii i postupci za pravljenje i upotrebu istih
ES2752191T3 (es) 2012-02-17 2020-04-03 Childrens Hospital Philadelphia Composiciones y métodos con vectores de AAV para la transferencia de genes a células, órganos y tejidos
CA2868391A1 (fr) * 2012-04-02 2013-10-10 Stephane Bancel Polynucleotides comprenant du n1-methyl-pseudouracils et methodes pour les preparer
GB201210357D0 (en) 2012-06-12 2012-07-25 Ucl Business Plc Factor VIII sequences
US8986991B2 (en) 2012-07-03 2015-03-24 Expression Therapeutics, Llc High yield suspension cell line, system and method for making same
WO2014064277A1 (fr) 2012-10-26 2014-05-01 Vrije Universiteit Brussel Vecteur pour thérapie génique ciblée sur le foie de l'hémophilie et procédés associés
EP3513802B1 (fr) 2012-10-26 2023-11-29 Vrije Universiteit Brussel Vecteur pour thérapie génique de l'hémophilie ciblée sur le foie et ses procédés et son utilisation
HRP20231183T1 (hr) 2013-02-15 2024-01-05 Bioverativ Therapeutics Inc. Optimizirani gen faktora viii
CA3237630A1 (fr) 2014-08-13 2016-02-18 The Children's Hospital Of Philadelphia Cassette d'expression amelioree pour le conditionnement et l'expression de variantes du facteur viii pour le traitement des troubles de l'hemostase
AU2016232146B2 (en) 2015-03-17 2021-11-04 Vrije Universiteit Brussel Optimized liver-specific expression systems for FVIII and FIX
BR112018009717B1 (pt) 2015-11-13 2021-12-14 Takeda Pharmaceutical Company Limited Polinucleotídeo, vetor de vírus adeno-associado, partícula de um vírus adeno-associado, métodos para produzir uma partícula de vírus adeno-associado e para transduzir uma célula hospedeira, e, uso de uma partícula de vírus adeno-associado

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20150071883A1 (en) * 2013-09-12 2015-03-12 Biomarin Pharmaceutical Inc. Adeno-Associated Virus Factor VIII Vectors

Also Published As

Publication number Publication date
BR112018009732A8 (pt) 2019-02-26
IL259295A (en) 2018-07-31
EA202092049A1 (ru) 2020-11-10
JP2018537089A (ja) 2018-12-20
JP6695426B2 (ja) 2020-05-20
WO2017083764A1 (fr) 2017-05-18
AU2016354550A1 (en) 2018-06-07
NZ742555A (en) 2023-12-22
SG11201804064WA (en) 2018-06-28
EP3374388A1 (fr) 2018-09-19
CL2020001611A1 (es) 2020-10-30
AU2016354550B2 (en) 2020-04-16
US20170226188A1 (en) 2017-08-10
CA3005565A1 (fr) 2017-05-18
CO2018005380A2 (es) 2018-05-31
EA201891138A1 (ru) 2018-12-28
IL259295B1 (en) 2024-06-01
CL2018001301A1 (es) 2018-12-28
BR112018009732A2 (pt) 2018-11-21
IL259295B2 (en) 2024-10-01
CN108602876B (zh) 2022-07-05
EA036944B1 (ru) 2021-01-19
KR102404550B1 (ko) 2022-05-31
TW202229555A (zh) 2022-08-01
ZA201803442B (en) 2019-07-31
MX2018005969A (es) 2018-11-29
CN108602876A (zh) 2018-09-28
US10189889B2 (en) 2019-01-29
TW201723180A (zh) 2017-07-01
KR20180070700A (ko) 2018-06-26

Similar Documents

Publication Publication Date Title
JP7307836B2 (ja) 組換えｆｖｉｉｉ変異型をコードする血友病ａ遺伝子治療用高発現ウイルスベクター
TWI823830B (zh) 用於ａ型血友病基因治療之編碼表現增加之重組ｆｖｉｉｉ變異體之病毒載體
CA3127065A1 (fr) Vecteurs viraux codant pour des variants fviii de recombinaison avec une expression accrue pour la therapie genique de l'hemophilie a
US12091675B2 (en) Gene therapy of hemophilia a using viral vectors encoding recombinant FVIII variants with increased expression
US20230023826A1 (en) Gene therapy of hemophilia a using viral vectors encoding recombinant fviii variants with increased expression
EA047630B1 (ru) Вирусные векторы, кодирующие рекомбинантные варианты fviii с повышенной экспрессией для генной терапии гемофилии а
EA046432B1 (ru) Вирусные векторы, кодирующие рекомбинантные варианты fviii с повышенной экспрессией для генной терапии гемофилии a
EA047302B1 (ru) Вирусные векторы, кодирующие рекомбинантные варианты fviii с повышенной экспрессией для генной терапии гемофилии a